Donate

Industry News

Genentech Meets Primary Endpoint for Phase III Emicizumab Study

December 22, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Genentech. Read the full press release in it鈥檚 entirety聽here. Genentech, a member of the Roche Group, announced that the primary endpoint has been met for the Phase III HAVEN 1 study evaluating emicizumab prophylaxis in people 12 years of age or older with hemophilia […]

UniQure Announces New Data in Phase I/II Clinical Trial

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from UniQure. Read the full press release in it鈥檚 entirety here. UniQure announced new and updated results during the American Society of Hematology (ASH) meeting聽in relation to its ongoing, dose-ranging Phase I/II trial of AMT-060. AMT-060 is UniQure鈥檚 proprietary, investigational gene therapy聽for patients with severe […]

Sangamo Biosciences Announces Plans to File for New Investigational Drug

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Sangamo Biosciences. Read the full press release in its entirety here. Sangamo Biosciences presented data during the recent American Society of Hematology (ASH) Annual Meeting regarding preclinical and manufacturing data of SB-525, its gene therapy program for hemophilia A. “We have developed an improved […]

Spark Therapeutics Shares Updated Phase I/II Clinical Trial Data

December 12, 2016

______________________________________________________________________________ Note: The following is edited from a press release from Spark Therapeutics. Read the full press release in it鈥檚 entirety here.. Spark Therapeutics and Pfizer announced updated results from the Phase 1/2 study of SPK-9001, an investigational recombinant gene transfer product in hemophilia B, at the 58th American Society of Hematology (ASH) Annual Meeting, […]

Alnylam Presents Phase I Clinical Drug Trial Data at ASH 2016

December 12, 2016

聽 Note: The following is an excerpt from a press release from Alnylam Pharmaceuticals, Inc. regarding news from their recent presentation at the American Society of Hematology (ASH) Annual Meeting. The original article can be read in its entirety here. ______________________________________________________________________________ Alnylam Pharmaceuticals, Inc. presented data from the phase 1 study of fitusiran at the […]

Octapharma Announces Two Hemophilia A Clinical Trials Utilizing Wilate

November 16, 2016

The following is excerpted from a press release from Octapharma USA. Read the press release in it’s entirety here.聽 Octapharma USA has announced that WILATE庐, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection will be utilized for two important clinical trials for hemophilia A launching in December. Octapharma USA […]

Prescription Benefit Program Changes for Recombinant Clotting Factor Product

October 18, 2016

  It has come to our attention that Helixate FS, a recombinant clotting factor product sold and distributed by CSL Behring, is being removed from prescription benefit programs for a limited number of聽patients using CVS/Caremark effective January 1, 2017. Patients currently using Helixate FS who have the “Advanced Control Formulary” plan with聽CVS/Caremark will have the […]

PPTA Releases Updated Statement on Zika

September 13, 2016

The following is a summary of a statement released by the Plasma Protein Therapeutics Association (PPTA).聽To read the PPTA statement in full, click聽HERE. On September 8, 2016, PPTA聽issued an updated聽news release聽regarding the Zika virus (ZIKV) and plasma protein therapies. In the release, PPTA notes that: ZIKV is in a family of (lipid-enveloped) viruses that is […]

Sangamo BioSciences Receives Orphan Drug Designation From The FDA For SB-FIX

September 9, 2016

The following is excerpted from a press release from PRNewswire. Read the press release in it’s entirety聽here. Sangamo BioSciences, Inc.聽announced earlier this week that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SB-FIX, the company’s zinc finger nuclease (ZFN)-mediated genome editing product candidate for the treatment of hemophilia B. Sangamo […]

Baxalta Sponsors 63 Students in 2016-17

August 22, 2016

Baxalta, now part of Shire, has awarded scholarships to 63 students from 13 states, further adding to the list of recipients of its Education Advantage scholarship program. For the seventh consecutive year, Baxalta US Inc. is helping eligible students with a bleeding disorder offset the costs of higher education, advance their careers, and pursue their […]

First FDA-Approved Recombinant von Willebrand Factor, Vonvendi, Now Available for von Willebrand Disease

August 9, 2016

The following is excerpted from a press release from Baxalta, now a part of Shire. Read the press release in it’s entirety here. Shire has announced that Vonvendi [von Willebrand factor (Recombinant)], indicated for the on-demand treatment and control of bleeding episodes in adults (age 18 and older) diagnosed with von Willebrand disease, is now […]

Biogen Names New Spin Off, Bioverativ; Aiming for Early 2017 Launch

August 9, 2016

The following is an excerpt from FierceBiotech.com To read the article in full, click聽here. Biogen previously announced its plan to spin out its blood disorder products and candidates into a separate publicly traded company. The new company will be known as Bioverativ and is slated to launch in early 2017 and trade on the Nasdaq […]

Shire Discontinues Hemophilia Gene Therapy Drug Development

August 4, 2016

The following is an excerpt from xconomy.com. To read the article in full, click here. On a conference call Tuesday, Shire聽CEO Flemming Ornskov said that the Dublin and Lexington, MA, company has discontinued development of BAX 335, an experimental gene therapy for hemophilia B.聽Prior to the merger of Baxalta and Shire, Baxalta had indicated plans […]

Genentech Presents Latest Data on Emicizumab (ACE910)

July 29, 2016

Note: The below is an edited version of an article originally published on BusinessWire.聽 The original article can be read in their entirety聽here. The latest data from an ongoing Japanese Phase I/II study of Emicizumab were presented earlier this week at the World Federation of Hemophilia 2016 World Congress.聽 Emicizumab (a product being developed by […]

BioMarin Releases Results of Phase 1/2 Study of Gene Therapy Treatment

July 28, 2016

Note: The below is an edited version of a press release issued by BioMarin Pharmaceutical Inc. The original release can be read in its entirety聽here. BioMarin Pharmaceutical announced today positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. Nine patients with severe hemophilia […]

Sangamo BioSciences Announces New Gene Therapy Clinical Development Program

July 28, 2016

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in its聽entirety聽here. Sangamo BioSciences, Inc. announced the presentation of preclinical data that supports the clinical development of its new proprietary gene therapy for the treatment of hemophilia A. This new therapeutic comprises an adeno-associated […]

uniQure Presents Updated Clinical Data in Patients with Severe Hemophilia B

July 28, 2016

Note: The below is an edited version of an article originally published on Globe Newswire. The original release can be read in its entirety聽here. uniQure N.V. yesterday announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, in patients with severe hemophilia B.聽 The updated data show that […]

CSL Behring Presents Phase 3 Data about Afstyla for Hemophilia A

July 27, 2016

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in it’s entirety聽here. CSL Behring聽today presented data from a Phase III pivotal study of Afstyla聽[Antihemophilic Factor (Recombinant), Single Chain] in hemophilia A patients less than 12 years of age at the XXXII International Congress […]

Voluntary Withdrawals of Helixate FS & Kogenate FS

July 26, 2016

As part of HFA鈥檚 ongoing mission to assist and advocate for the bleeding disorders community, this medical news is being issued to keep you informed about product safety. About These Withdrawals CSL Behring is initiating a voluntarily recall of two lots of its hemophilia A drug Helixate FS containing active ingredient manufactured before November 2015. […]

Adynovate Phase 3 Efficacy and Safety Data in Children to be Presented

July 26, 2016

Note: The below is an edited version of an article originally published on PR Newswire. The original release can be read in it’s entirety here. _________________________________________________________________________________________ Shire will present additional data on the clinical experience of ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended half-life recombinant Factor VIII (rFVIII) replacement for hemophilia A, during the International […]

CSL Behring Presents Phase III Data for IDELVION for Hemophilia B

July 26, 2016

Note: The below is an edited version of an article originally published on Yahoo Finance. The original release can be read in it’s entirety here. _________________________________________________________________________________________ CSL Behring announced new data today from its Phase III PROLONG-9FP clinical development program evaluating聽IDELVION庐 [Coagulation Factor IX (Recombinant), Albumin Fusion Protein], the company’s long-acting recombinant albumin fusion protein […]

Alnylam Announces New Positive Interim Phase 1 Study Results for Fitusiran

July 26, 2016

Note: The below is an edited version of an article originally published on StreetInsider.com. The original release can be read in it’s entirety聽here. _________________________________________________________________________________________ Alnylam Pharmaceuticals, Inc. announced today new positive results from its ongoing Phase 1 study with Fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia A and B and […]

Novo Nordisk Announces New Data on Psychosocial Impact of People Living with Hemophilia B

July 26, 2016

Note: The below is an edited version of a press release by Novo Nordisk. The original release can be read in it’s entirety聽here.聽 Novo Nordisk today announced the first results from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study, which found that many adults and children living with even mild or […]

Spark Therapeutics and Pfizer Announce Receipt of FDA Breakthrough Therapy Designation for SPK-9001 for the Treatment of Hemophilia B

July 21, 2016

Note: The below is an edited version of a press release by Spark Therapeutics.聽The original release can be read in it’s entirety聽here. Spark Therapeutics and Pfizer Inc. announced today that the聽U.S. Food and Drug Administration聽(FDA) has granted breakthrough therapy designation to聽SPK-9001,聽the lead investigational candidate in the companies’聽SPK-FIX聽program, in development for the treatment of hemophilia B.聽SPK-9001,聽a […]

Kedrion Announces Voluntary, Temporary Hold on Koate-DVI

July 6, 2016

Kedrion Biopharma provided the following announcement today: Koate庐-DVI Summer Supply Statement Kedrion Biopharma has placed a voluntary, temporary hold on further shipments聽of Koate-DVI until approximately the September timeframe. The company is working to restore supplies of Koate-DVI as soon as possible so聽that it can be made available to patients who require it. This is being […]

uniQure Announces Data from Phase I/II AMT-060 Clinical Trial

June 28, 2016

Note: The below is an edited version of a press release by UniQure.聽The original release can be read in it’s entirety聽here. On June 11, 2016,聽uniQure N.V. announced that additional data from its Phase I/II clinical trial of AMT-060 in hemophilia B patients were presented at the 21st聽Congress of the European Hematology Association (EHA) in Copenhagen, […]

Spark Therapeutics Announces Updated Data from Hemophilia B Trial

June 14, 2016

Note: The below is an edited version of a press release by Spark Therapeutics.聽The original release can be read in it’s entirety聽here. PHILADELPHIA,聽June 13, 2016 –聽Spark Therapeutics announced today updated results of the first cohort from the ongoing Phase 1/2 clinical trial of聽SPK-9001, the lead investigational candidate in its聽SPK-FIX聽program, which is being studied for the […]

Shire Completes Combination with Baxalta

June 3, 2016

Today, June 3, 2016, Shire completed its previously announced acquisition with Baxalta Incorporated. The combined company is expected to deliver over $20 billion in annual revenues by 2020. The聽company is focused on serving patients with rare diseases and other highly specialized conditions. Read the press release from Shire in it’s entirety here.   About Shire聽 […]

FDA Approves First Single Chain Product for Hemophilia A

May 27, 2016

Note: The below is an edited version of a press release by CSL Behring.聽The original release can be read in it’s entirety聽here. KING OF PRUSSIA, Pa., May 26, 2016 — CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved AFSTYLA [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor […]

Biogen Announces Intent to Spin off Its Hemophilia Business

May 3, 2016

Note: The below is an edited version of a press release from Biogen Inc. The original release can be read in its entirety here. Biogen Inc. announced on Tuesday, May 3, 2016 that it intends to spin off its hemophilia business as an independent, publicly traded company. The strategic goal of this transaction is to […]


Sign up for E-mails, Dateline Magazine, and other ways to stay connected.